Angelo Lombardo

Summary

Affiliation: Federico II University
Country: Italy

Publications

  1. ncbi request reprint Identification of molecular defects in Italian Sanfilippo A patients including 13 novel mutations
    P Di Natale
    Dipartimento di Biochimica e Biotecnologie Mediche, Medical School, University of Naples Federico II, Italy
    Hum Mutat 11:313-20. 1998
  2. ncbi request reprint Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery
    Angelo Lombardo
    San Raffaele Telethon Institute for Gene Therapy, Via Olgettina, 58, 20132 Milan, Italy
    Nat Biotechnol 25:1298-306. 2007
  3. ncbi request reprint Heparan N-sulfatase: in vitro mutagenesis of potential N-glycosylation sites
    P Di Natale
    Department of Biochemistry and Medical Biotechnologies, University of Naples Federico II, Naples, Italy
    Biochem Biophys Res Commun 280:1251-7. 2001
  4. ncbi request reprint In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectors
    Paola Di Natale
    Department of Biochemistry and Medical Biotechnologies, University of Naples Federico II, Italy
    Eur J Biochem 269:2764-71. 2002
  5. ncbi request reprint Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector
    Carmela Di Domenico
    Department of Biochemistry and Medical Biotechnologies, University of Naples Federico II, 80131 Naples, Italy
    Hum Gene Ther 16:81-90. 2005
  6. ncbi request reprint Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice
    Antonia Follenzi
    Laboratory of Gene Transfer and Therapy, Institute for Cancer Research and Treatment IRCC, University of Torino, Torino, Italy
    Blood 103:3700-9. 2004
  7. ncbi request reprint The immune response to lentiviral-delivered transgene is modulated in vivo by transgene-expressing antigen-presenting cells but not by CD4+CD25+ regulatory T cells
    Andrea Annoni
    San Raffaele Telethon Institute for Gene Therapy HSR TIGET, Milan, Italy
    Blood 110:1788-96. 2007
  8. ncbi request reprint A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice
    Brian D Brown
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy
    Blood 110:4144-52. 2007
  9. ncbi request reprint Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Nonobese diabetic/severe combined immunodeficient
    Wanda Piacibello
    Department of Oncological Sciences, University of Torino Medical School, Torino, Italy
    Blood 100:4391-400. 2002

Collaborators

Detail Information

Publications9

  1. ncbi request reprint Identification of molecular defects in Italian Sanfilippo A patients including 13 novel mutations
    P Di Natale
    Dipartimento di Biochimica e Biotecnologie Mediche, Medical School, University of Naples Federico II, Italy
    Hum Mutat 11:313-20. 1998
    ..Interestingly, all six patients from Sardinia present this mutation, and five of them are homozygous for this change, suggesting that these subjects may have been derived from a common founder...
  2. ncbi request reprint Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery
    Angelo Lombardo
    San Raffaele Telethon Institute for Gene Therapy, Via Olgettina, 58, 20132 Milan, Italy
    Nat Biotechnol 25:1298-306. 2007
    ....
  3. ncbi request reprint Heparan N-sulfatase: in vitro mutagenesis of potential N-glycosylation sites
    P Di Natale
    Department of Biochemistry and Medical Biotechnologies, University of Naples Federico II, Naples, Italy
    Biochem Biophys Res Commun 280:1251-7. 2001
    ..These studies confirm that the five glycosylation sites of heparan N-sulfatase are all functional and show that Asn 41 and Asn 151 have a role in protein folding and/or stability...
  4. ncbi request reprint In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectors
    Paola Di Natale
    Department of Biochemistry and Medical Biotechnologies, University of Naples Federico II, Italy
    Eur J Biochem 269:2764-71. 2002
    ..These results suggest that the lentiviral vector may be used for the delivery and expression of the IDUA gene to cells in vivo for treatment of MPS I...
  5. ncbi request reprint Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector
    Carmela Di Domenico
    Department of Biochemistry and Medical Biotechnologies, University of Naples Federico II, 80131 Naples, Italy
    Hum Gene Ther 16:81-90. 2005
    ..In conclusion, our results show the promising potential and the limitations of lentiviral-IDUA vector-mediated gene therapy in an in vivo model...
  6. ncbi request reprint Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice
    Antonia Follenzi
    Laboratory of Gene Transfer and Therapy, Institute for Cancer Research and Treatment IRCC, University of Torino, Torino, Italy
    Blood 103:3700-9. 2004
    ..These results prompt further studies in relevant animal models to explore the potential of in vivo LV administration for the gene therapy of hemophilias and other liver-based diseases...
  7. ncbi request reprint The immune response to lentiviral-delivered transgene is modulated in vivo by transgene-expressing antigen-presenting cells but not by CD4+CD25+ regulatory T cells
    Andrea Annoni
    San Raffaele Telethon Institute for Gene Therapy HSR TIGET, Milan, Italy
    Blood 110:1788-96. 2007
    ..These data indicate that antitransgene immune response can be modulated by transgene-expressing APCs possibly through deletion of effector T cells...
  8. ncbi request reprint A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice
    Brian D Brown
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy
    Blood 110:4144-52. 2007
    ..This work, which is among the first applications to exploit the microRNA regulatory pathway, provides the basis for a promising new therapy for the treatment of hemophilia B...
  9. ncbi request reprint Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Nonobese diabetic/severe combined immunodeficient
    Wanda Piacibello
    Department of Oncological Sciences, University of Torino Medical School, Torino, Italy
    Blood 100:4391-400. 2002
    ..8% +/- 5.9% of human cells were GFP(+), and human engraftment was multilineage. These results show that lentiviral vectors efficiently transduce HSCs, which can undergo expansion and maintain proliferation and self-renewal ability...